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Development of an AAV-based gene therapy for the ocular phenotype of Friedreich's ataxia.

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Title: Development of an AAV-based gene therapy for the ocular phenotype of Friedreich's ataxia.
Authors: Tang H; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Gupte S; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Xu E; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Calabro KR; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Friend H; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Crosson SM; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Fajardo D; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Kostamo Z; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Zhang H; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Peterson JJ; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Lin F; Department of Ophthalmology, Emory University, Atlanta, GA, USA., Kozmik Z; Institute of Molecular Genetics, Academy of Sciences of the Czech Republic, Prague, Czech Republic., Lutz CM; Rare and Orphan Disease Center, The Jackson Laboratory, Bar Harbor, ME, USA., Boye SL; Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA., Boye SE; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA. Electronic address: shaire@ufl.edu.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2026 Feb 04; Vol. 34 (2), pp. 771-788. Date of Electronic Publication: 2025 Oct 24.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Development of an AAV-based gene therapy for the ocular phenotype of Friedreich's ataxia.
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  Data: <searchLink fieldCode="AU" term="%22Tang+H%22">Tang H</searchLink>; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Gupte+S%22">Gupte S</searchLink>; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Xu+E%22">Xu E</searchLink>; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Calabro+KR%22">Calabro KR</searchLink>; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Friend+H%22">Friend H</searchLink>; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Crosson+SM%22">Crosson SM</searchLink>; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Fajardo+D%22">Fajardo D</searchLink>; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Kostamo+Z%22">Kostamo Z</searchLink>; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Zhang+H%22">Zhang H</searchLink>; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Peterson+JJ%22">Peterson JJ</searchLink>; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Lin+F%22">Lin F</searchLink>; Department of Ophthalmology, Emory University, Atlanta, GA, USA.<br /><searchLink fieldCode="AU" term="%22Kozmik+Z%22">Kozmik Z</searchLink>; Institute of Molecular Genetics, Academy of Sciences of the Czech Republic, Prague, Czech Republic.<br /><searchLink fieldCode="AU" term="%22Lutz+CM%22">Lutz CM</searchLink>; Rare and Orphan Disease Center, The Jackson Laboratory, Bar Harbor, ME, USA.<br /><searchLink fieldCode="AU" term="%22Boye+SL%22">Boye SL</searchLink>; Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Boye+SE%22">Boye SE</searchLink>; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA. Electronic address: shaire@ufl.edu.
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              Text: 2026 Feb 04
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